Translational Research Center to Expedite Novel Therapies in Cystic Fibrosis

转化研究中心将加快囊性纤维化的新疗法

基本信息

批准号：
9762084
负责人：
Bonnie W Ramsey
金额：
$ 121.3万
依托单位：
SEATTLE CHILDREN'S HOSPITAL
依托单位国家：
美国
项目类别：
财政年份：
2010
资助国家：
美国
起止时间：
2010-07-06 至 2020-05-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/9762084
关键词：
Acute Affect Antibiotic Therapy Area Basic Science Biological Biological Assay Biological Markers Cell physiology Cells Center for Translational Science Activities Child Chronic lung disease Clinical Clinical Microbiology Clinical Research Clinical Trials Clinical Trials Design Collaborations Communities Cystic Fibrosis Cystic Fibrosis Transmembrane Conductance Regulator DNA Data Development Digestive System Disorders Direct Costs Disease Disease Progression Doctor of Philosophy Drug Screening Ecosystem Environmental Risk Factor Epithelial Epithelial Cells Fostering Functional disorder Funding Gastroenterology Gastrointestinal tract structure Genetic Diseases Genomics Grant Health Hepatobiliary Human Immune Immune response Immunology Infant Infection Inflammation Inflammatory Infrastructure Inhalation Institution Interdisciplinary Study International Intestines Islet Cell Kidney Laboratories Leadership Link Lung Lung infections Malnutrition Measures Medicine Metabolic Metabolism Methodology Microbiology Mutation National Institute of Diabetes and Digestive and Kidney Diseases Nutritional Observational Study Outcome Outcome Measure Pancreas Pathogenesis Patients Pediatrics Pharmaceutical Preparations Physiological Process Proteins Pseudomonas aeruginosa Recording of previous events Research Research Institute Research Personnel Resources Respiratory System Scientist Specimen Testing Tissues Tobramycin Translational Research Translations United States National Institutes of Health Universities Vision Washington base children with cystic fibrosis clinical database comparative comparative effectiveness cystic fibrosis patients data resource design drug development effectiveness research experience genome sciences gut microbiome improved in vivo interest medical schools microbiome research mid-career faculty next generation novel therapeutic intervention novel therapeutics nutrition professor programs public health relevance ranpirnase repository response small molecule therapeutic development

项目摘要

DESCRIPTION (provided by applicant): This P30 Cystic Fibrosis (CF) Research and Translational Core Center (CFRTC) is located at the University of Washington School of Medicine (UWSOM) and its affiliated institution, Seattle Children's Research Institute (SCRI). This proposed P30 center program enhances an existing robust basic and clinical research base at these institutions totally over $20 million direct costs in current research funding and an additional $5 million in CF related infrastructure funding, and includes 48 UWSOM investigators in 19 departments and/or divisions. The UWSOM has a long history of excellence in CF related translational research as evidenced by the successful development of inhaled tobramycin (TOBI(r)) and the leadership in the CFF supported Therapeutics Development Network. The research for this P30 will focus on four Specific Aims: 1) Provide resources and expertise to expedite development of promising new therapeutic approaches to correct dysfunctional CFTR and its secondary consequences including maldigestion and malnutrition, hepatobiliary disease and chronic lung disease. 2) Foster collaboration across P-30 biomedical cores, nationally and internationally to enhance translational research through sharing of human and bacterial repositories linked to clinical databases. Utilize these shared specimen and data resources to develop improved assays, biomarkers and clinical outcome measures. 3) Enhance understanding of the evolving bacterial ecosystems and resultant host response in the gastrointestinal and respiratory tracts of patients with cystic fibrosis and how these interactions impact health. 4) Better understand the metabolic and inflammatory consequences of CFTR dysfunction. The center will be led by Co-PD's, Bonnie Ramsey, MD and E. Peter Greenberg PhD, CF investigators both internationally recognized in their respective areas of clinical research and bacterial pathogenesis. They will be supported by other key leadership: Samuel Miller MD, Professor of Medicine, Microbiology, Genome Sciences, and Immunology, Charles Frevert, DVM, ScD, Associate Professor in the Department of Comparative Medicine, Christopher Goss MD, MPH, Professor of Medicine and Pediatrics, and Pradeep Singh MD, Professor of Medicine. The center will consist of an Administrative Core (Ramsey and Greenberg, Directors) and four biomedical cores, Microbiology (Greenberg, Director), Genomics (Miller, Director), Host Response (Frevert, Director) and Clinical Translational (Christopher Goss and Ramsey, Directors). The core centers will operate a Pilot and Feasibility (P&F) program led by Dr. Pradeep Singh. Three newly approved P&F projects are included in the current proposal chosen through a competitive process and approved by the Internal Advisory Board. During the funding period, the core center will focus on development of several novel therapeutic approaches to improve overall health for patients with CF.

描述（由申请人提供）：此P30囊性纤维化（CF）研究与转化核心中心（CFRTC）位于华盛顿大学医学院（UWSOM）及其附属机构，西雅图儿童研究所（SCRI）。该拟议的P30中心计划在这些机构中增强了现有的强大基础和临床研究基础，目前的研究资金全额超过2000万美元的直接成本，另外还有500万美元的CF相关基础设施资金，其中包括19个部门和/或部门的48位UWSOM调查人员。 UWSOM在CF相关的转化研究方面拥有悠久的卓越历史，这是吸入毒素（Tobi（R））的成功开发和CFF支持的治疗学发展网络的领导能力所证明的。该P30的研究将重点放在四个具体目标上：1）提供资源和专业知识，以加快有希望的新治疗方法来纠正功能障碍的CFTR及其次要后果，包括Maldigestion和营养不良，肝疾病和肝病。 2）在国内和国际上培养跨P-30生物医学核心的合作，以通过共享与临床数据库相关的人类和细菌存储库来增强转化研究。利用这些共享标本和数据资源来开发改进的测定法，生物标志物和临床结果指标。 3）增强对不断发展的细菌生态系统的了解以及囊性纤维化患者胃肠道和呼吸道中的宿主反应以及这些相互作用如何影响健康。 4）更好地了解CFTR功能障碍的代谢和炎症后果。该中心将由CO-PD，Bonnie Ramsey，MD和E. Peter Greenberg博士领导，CF研究人员都在其各自的临床研究和细菌发病机理的领域都在国际上认可。他们将得到其他主要领导力的支持：塞缪尔·米勒（Samuel Miller），医学，微生物学，基因组科学和免疫学教授，查尔斯·弗雷弗特（Charles Frevert），DVM，SCD，比较医学系副教授，克里斯托弗·戈斯（Christopher Goss），医学和儿童医学和儿童医学和Pradeep Singh MD MD，MDICH MD，MEDH，MEDH MD，MEDH，MEDH，MEDER，MEDH MD，MEDH。该中心将由行政核心（Ramsey and Greenberg，董事）和四个生物医学核心，微生物学（Greenberg，董事），基因组学（Miller，Director），主机反应（FREVERT，董事）和临床转化（Christopher Goss和Ramsey，导演）。核心中心将运营由Pradeep Singh博士领导的飞行员和可行性（P＆F）计划。通过竞争过程选择的当前提案中包括三个新批准的P＆F项目，并由内部咨询委员会批准。在资助期间，核心中心将重点放在开发几种新型治疗方法，以改善CF患者的整体健康状况。